Cystic Fibrosis: VX-121/Tezacaftor/Deutivacaftor Therapy

We are studying a new combination therapy for children with cystic fibrosis to evaluate its safety and how well it works. This trial focuses on kids aged 1 to 11 years.

What diseases are being studied?

Health conditions and diseases that the clinical trial is designed to study and treat.

Who can participate in the clinical trial?

These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..

What medicines are being studied?

Medicines that are officially approved for use in the European Union and considered safe by EMA. These medicines are already on the market and may be tested for new conditions or populations.

Kaftrio
Kaftrio is a medicine used to treat cystic fibrosis by improving lung function and reducing related symptoms.
Kalydeco
Kalydeco is a medicine for people with certain forms of cystic fibrosis that helps improve lung function and reduce symptoms.

What active substances are being studied?

Active substances that are officially approved for use in the European Union and considered safe by EMA. These substances are already on the market and may be tested for new conditions or populations.

Elexacaftor
Elexacaftor is a substance that improves function of the defective protein in cystic fibrosis to help lungs and other organs work better.
Ivacaftor
Ivacaftor is a substance that improves a faulty protein in people with certain types of cystic fibrosis to help breathing and lung function.
Tezacaftor
Tezacaftor is a substance that helps correct a faulty protein in cystic fibrosis, improving lung function and reducing mucus buildup.

What experimental substances are being tested?

Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.

Vanzacaftor
Deutivacaftor

What similar medicines might you know?

Don't worry if you don't know the medicines tested in the trial. There is a chance you have heard about the similar medicines. Check the list below to see if you are familiar with any of them.

Symkevi

Where is the clinical trial being conducted?

Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.

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Hospital Femme Mere Enfant
Service de Pédiatrie, Allergologie, Mucoviscidose
Bron, France
Hopital Necker Enfants Malades
Hôpital Necker - Enfants Malades, Service de Pneumologie et Allergologie Pédiatriques
Montrouge, France
Charite Universitaetsmedizin Berlin KöR
Department of Pediatric Pulmonology Immunology and Critical Care Medicine
Berlin, Germany

Sponsor: Vertex Pharmaceuticals Inc.
Last updated: Nov 26, 2025

Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.